Alpha-1 Antitrypsin Deficiency (AATD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Alpha-1 antitrypsin deficiency (AATD) Market Outlook and Forecast

Alpha-1 Antitrypsin Deficiency (AATD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Published Date : 2021-11-25

Updated On : 2023-09-04

Pages : 160

Alpha-1 Antitrypsin Deficiency (AATD) Market Outlook

Thelansis’s “Alpha-1 Antitrypsin Deficiency (AATD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Alpha-1 Antitrypsin Deficiency treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Alpha-1 Antitrypsin Deficiency (AATD) Overview

Alpha-1 Antitrypsin Deficiency (AATD) is a hereditary condition, often overlooked, characterized by low circulating alpha-antitrypsin (AAT) in the bloodstream. This condition presents as hepatic dysfunction from infancy through adulthood and chronic obstructive lung disease (specifically emphysema or bronchiectasis) in individuals over 30. People with AATD also face an elevated risk of panniculitis and C-ANCA-positive vasculitis. The expression of these symptoms can vary among individuals and families. In adults, smoking is the primary factor accelerating the development of chronic obstructive pulmonary disease (COPD). However, nonsmokers with AATD can still develop lung and liver issues and have an average life expectancy. Emphysema in children with AATD is exceedingly rare. AATD-related liver disease, seen in only a small fraction of affected children, typically presents as neonatal cholestasis. The incidence of liver disease increases with age, and adults may develop cirrhosis and fibrosis without a history of neonatal or childhood liver issues. Additionally, individuals with AATD are at an increased risk of hepatocellular carcinoma (HCC).

  • The prevalence of alpha-1 Antitrypsin Deficiency (AATD) ranges from 1 to 2 cases per 10,000 population in the USA. 

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2021-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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Alpha-1 Antitrypsin Deficiency (AATD) Competitive Landscape

S. no Asset Company Stage
1 Fazirsiran Injection Takeda Phase 3
2 Alpha-1 MP Grifols Therapeutics LLC Phase 3
3 ADVM-043 Adverum Biotechnologies, Inc. Phase 2
4 Alpha 1-Antitrypsin Kamada, Ltd. Phase 3
5 INBRX-101 Inhibrx, Inc. Phase 2
6 Fazisiran Injection Arrowhead Pharmaceuticals Phase 2
7 Alvelestat (MPH966) Mereo BioPharma Phase 2
8 VX-634 Vertex Pharmaceuticals Incorporated Phase 1
9 Belcesiran Dicerna Pharmaceuticals, Inc., a Novo Nordisk company Phase 2
10 Alpha-1 antitrypsin (AAT) CSL Behring Phase 3

Continued...

KOLs across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

COUNTRY No. Of KOLs
USA 17
GERMANY 4
UK 4
SPAIN 3
FRANCE 2
ITALY 3
JAPAN 3
CHINA 4

Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

Data Inputs with sourcing, Market Event, Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Alpha-1 antitrypsin deficiency (AATD) Market Forecast

1.       Alpha-1 Antitrypsin Deficiency (AATD) – Key Findings Summary

         1.1.    Clinical findings
                     1.1.1. Disease overview
                     1.1.2. Therapeutic practices
                     1.1.3. Future outlook
         1.2.    Commercial findings
                     1.2.1. Alpha-1 Antitrypsin Deficiency (AATD) market scenario 2021
                     1.2.2. Alpha-1 Antitrypsin Deficiency (AATD) market scenario 2025
                     1.2.3. Alpha-1 Antitrypsin Deficiency (AATD) market scenario 2032

2.       Alpha-1 Antitrypsin Deficiency (AATD) Overview

         2.1.    Disease Introduction
         2.2.    Pathophysiology
         2.3.    Signs and Symptoms
         2.4.    Risk Factors
         2.5.    Etiology
         2.6.    Classification
         2.7.    Pathogenesis
         2.8.    Diagnosis
         2.9.    Complications
         2.10. Treatment Algorithm
                     2.10.1.    Treatment in US (guidelines)
                     2.10.2.    Treatment in EU-5 (guidelines)
                     2.10.3.    Treatment in Japan (guidelines)
                     2.10.4.    Treatment in China (guidelines)
         2.11. Treatment Goals for Alpha-1 Antitrypsin Deficiency (AATD)
         2.12. Referral Patterns
                     2.12.1.    Referral Scenario in US
                     2.12.2.    Referral Scenario in EU-5
                     2.12.3.    Referral Scenario in Japan
                     2.12.4.    Referral Scenario in China
         2.13. Alpha-1 Antitrypsin Deficiency (AATD) Prognosis
         2.14. Healthcare burden
                     2.14.1.    Healthcare burden in US
                     2.14.2.    Healthcare burden in EU-5
                     2.14.3.    Healthcare burden in Japan
                     2.14.4.    Healthcare burden in China
         2.15.  Unmet Needs in Alpha-1 Antitrypsin Deficiency (AATD) management
         2.16.  Market Opportunity for Alpha-1 Antitrypsin Deficiency (AATD)
         2.17. KOL Comments on current and upcoming/expected treatment practices in Alpha-1 Antitrypsin Deficiency (AATD)

3.       Epidemiology

         3.1.    Epidemiology Overview
         3.2.    Epidemiology by Geography
                     3.2.1.  Alpha-1 Antitrypsin Deficiency (AATD) Epidemiology in US (2021-2032)
                             3.2.1.1.              Incidence of Alpha-1 Antitrypsin Deficiency (AATD)
                             3.2.1.2.              Diagnosed cases
                             3.2.1.3.              Treatable Patient Pool
                             3.2.1.4.              Epidemiology Trends
                     3.2.2.  Alpha-1 Antitrypsin Deficiency (AATD) Epidemiology in EU-5 (2021-2032)
                             3.2.2.1.              Incidence of Alpha-1 Antitrypsin Deficiency (AATD)
                             3.2.2.2.              Diagnosed cases
                             3.2.2.3.              Treatable Patient Pool
                             3.2.2.4.              Epidemiology Trends
                     3.2.3.  Alpha-1 Antitrypsin Deficiency (AATD) Epidemiology in Japan (2021-2032)
                             3.2.3.1.              Incidence of Alpha-1 Antitrypsin Deficiency (AATD)
                             3.2.3.2.              Diagnosed cases
                             3.2.3.3.              Treatable Patient Pool
                             3.2.3.4.              Epidemiology Trends
                     3.2.4.  Alpha-1 Antitrypsin Deficiency (AATD) Epidemiology in China (2021-2032)
                             3.2.4.1.              Incidence of Alpha-1 Antitrypsin Deficiency (AATD)
                             3.2.4.2.              Diagnosed cases
                             3.2.4.3.              Treatable Patient Pool
                             3.2.4.4.              Epidemiology Trends
         3.3.    Epidemiology Trends (World-wide)

4.       Market Outlook

         4.1.    US Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast 2021-2032
                     4.1.1. Market Progression (Futuristic)
                     4.1.2. Market Trends and Expectations
                             4.1.2.1.              Worst case scenario
                             4.1.2.2.              Base Case Scenario
                             4.1.2.3.              Best Case Scenario
                     4.1.3. Drivers and Barriers
         4.2.    UK Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast 2021-2032
                     4.2.1. Market Progression (Futuristic)
                     4.2.2. Market Trends and Expectations
                             4.2.2.1.              Worst case scenario
                             4.2.2.2.              Base Case Scenario
                             4.2.2.3.              Best Case Scenario
                     4.2.3. Drivers and Barriers
         4.3.    France Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast 2021-2032
                     4.3.1. Market Progression (Futuristic)
                     4.3.2. Market Trends and Expectations
                             4.3.2.1.              Worst case scenario
                             4.3.2.2.              Base Case Scenario
                             4.3.2.3.              Best Case Scenario
                     4.3.3. Drivers and Barriers
         4.4.    Germany Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast 2021-2032
                     4.4.1. Market Progression (Futuristic)
                     4.4.2. Market Trends and Expectations
                             4.4.2.1.              Worst case scenario
                             4.4.2.2.              Base Case Scenario
                             4.4.2.3.              Best Case Scenario
                     4.4.3. Drivers and Barriers
         4.5.    Italy Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast 2021-2032
                     4.5.1. Market Progression (Futuristic)
                     4.5.2. Market Trends and Expectations
                             4.5.2.1.              Worst case scenario
                             4.5.2.2.              Base Case Scenario
                             4.5.2.3.              Best Case Scenario
                     4.5.3. Drivers and Barriers
         4.6.    Spain Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast 2021-2032
                     4.6.1. Market Progression (Futuristic)
                     4.6.2. Market Trends and Expectations
                             4.6.2.1.              Worst case scenario
                             4.6.2.2.              Base Case Scenario
                             4.6.2.3.              Best Case Scenario
                     4.6.3. Drivers and Barriers
         4.7.    Japan Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast 2021-2032
                     4.7.1. Market Progression (Futuristic)
                     4.7.2. Market Trends and Expectations
                             4.7.2.1.              Worst case scenario
                             4.7.2.2.              Base Case Scenario
                             4.7.2.3.              Best Case Scenario
                     4.7.3. Drivers and Barriers
         4.8.    China Alpha-1 Antitrypsin Deficiency (AATD) Market Forecast 2021-2032
                     4.8.1. Market Progression (Futuristic)
                     4.8.2. Market Trends and Expectations
                             4.8.2.1.              Worst case scenario
                             4.8.2.2.              Base Case Scenario
                             4.8.2.3.              Best Case Scenario
                     4.8.3. Drivers and Barriers
         4.9.    Key Expected Milestones (world-wide) Impacting the Market

5.       Competitive Landscape

         5.1.    Pipeline Therapies Overview
                     5.1.1. Phase III Therapies
                                      5.1.1.1.                     Current Status
                                      5.1.1.2.                     Trial details, results
                                      5.1.1.3.                     Approval Timeline
                                      5.1.1.4.                     Likelihood of approval
                                      5.1.1.5.                     Expected Product Positioning
                             5.1.1.2.              All other Phase III Therapies …..
                             5.1.1.3.              Attribute Analysis of Phase III molecules
                     5.1.2.  Phase II and Phase I/II Therapies
                                      5.1.2.1.                     Current Status
                                      5.1.2.2.                     Trial details, results
                                      5.1.2.3.                     Approval Timelines
                     5.1.3. List of active Pre-clinical Therapies
                                      5.1.3.1.                     Status in Alpha-1 Antitrypsin Deficiency (AATD)
                                      5.1.3.2.                     Company positioning
                             5.1.3.2.              All other pre-clinical therapies
                     5.1.4. List of Inactive/discontinued assets
                             5.1.4.1.              Business impact of discontinuations on current pipeline
                     5.1.5. Potential winners from Alpha-1 Antitrypsin Deficiency (AATD) Pipeline
                             5.1.5.1.              Potential Blockbusters across the pipeline

6.       Regulatory/Approval Scenario

         6.1.    Regulatory/Approval Framework in US
                     6.1.1. Policy Framework
                     6.1.2. Payer Expectations
         6.2.    Regulatory/Approval Framework in UK
                     6.2.1. Policy Framework
                     6.2.2. Payer Expectations
         6.3.    Regulatory/Approval Framework in France
                     6.3.1. Policy Framework
                     6.3.2. Payer Expectations
         6.4.    Regulatory/Approval Framework in Germany
                     6.4.1. Policy Framework
                     6.4.2. Payer Expectations
         6.5.    Regulatory/Approval Framework in Italy
                     6.5.1. Policy Framework
                     6.5.2. Payer Expectations
         6.6.    Regulatory/Approval Framework in Spain
                     6.6.1. Policy Framework
                     6.6.2. Payer Expectations
         6.7.       Regulatory/Approval Framework in Japan
                     6.7.1. Policy Framework
                     6.7.2. Payer Expectations
         6.8.       Regulatory/Approval Framework in China
                     6.8.1. Policy Framework
                     6.8.2. Payer Expectations

7.       Clinical Trial Assessment – Current and Future Paradigm

         7.1.    Distribution of Primary Endpoints across trials
         7.2.    Distribution of Secondary Endpoints across trials
         7.3.    Evolution and acceptance of surrogate endpoints
         7.4.    Key Investigator initiated trials
         7.5.    Attrition analysis
                     7.5.1. Suspended/Discontinued Assets
                     7.5.2. Failed Trials, Reasons and Business Impact
                     7.5.3. Terminated Trials, Reasons and Business Impact
                     7.5.4. Withdrawn Trials, Reasons and Business Impact
         7.6.    Trial enrollment scenario and challenges
         7.7.    Clinical Trial Guidance (across geographies)

8.       Thelansis Commentary

         8.1.    Key Unmet needs in Alpha-1 Antitrypsin Deficiency (AATD)
         8.2.    Possible Best-case Clinical Trial Strategies
         8.3.    Possible Best Case Targeted Product Profile (TPP)
         8.4.    Possible Best-case Market positioning strategies
         8.5.    Possible Best-case Market Access Strategies
         8.6.    Possible Best-case LCM Strategies
         8.7.    Overall View on Alpha-1 Antitrypsin Deficiency (AATD) Market in Dollar Value

9.       Report Methodology

         9.1.    Secondary research
         9.2.    Primary research
         9.3.    Data collation
         9.4.    Insight Generation

10.   About Thelansis

         10.1.    Our Capabilities
         10.2.    Our Services
         10.3.    Our Contacts
         10.4.    Disclaimer